JP2021515548A5 - - Google Patents

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Publication number
JP2021515548A5
JP2021515548A5 JP2020545159A JP2020545159A JP2021515548A5 JP 2021515548 A5 JP2021515548 A5 JP 2021515548A5 JP 2020545159 A JP2020545159 A JP 2020545159A JP 2020545159 A JP2020545159 A JP 2020545159A JP 2021515548 A5 JP2021515548 A5 JP 2021515548A5
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JP
Japan
Prior art keywords
seq
capsid
acid sequence
proteins
amino acid
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JP2020545159A
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English (en)
Japanese (ja)
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JP2021515548A (ja
JPWO2019169004A5 (https=
JP7498665B2 (ja
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Priority claimed from PCT/US2019/019861 external-priority patent/WO2019169004A1/en
Publication of JP2021515548A publication Critical patent/JP2021515548A/ja
Publication of JP2021515548A5 publication Critical patent/JP2021515548A5/ja
Publication of JPWO2019169004A5 publication Critical patent/JPWO2019169004A5/ja
Priority to JP2024088641A priority Critical patent/JP2024115558A/ja
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Publication of JP7498665B2 publication Critical patent/JP7498665B2/ja
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JP2020545159A 2018-02-27 2019-02-27 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用 Active JP7498665B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024088641A JP2024115558A (ja) 2018-02-27 2024-05-31 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用

Applications Claiming Priority (29)

Application Number Priority Date Filing Date Title
US201862635968P 2018-02-27 2018-02-27
US201862635964P 2018-02-27 2018-02-27
US62/635,964 2018-02-27
US62/635,968 2018-02-27
US201862663788P 2018-04-27 2018-04-27
US201862663797P 2018-04-27 2018-04-27
US62/663,788 2018-04-27
US62/663,797 2018-04-27
US201862667587P 2018-05-06 2018-05-06
US62/667,587 2018-05-06
US201862667881P 2018-05-07 2018-05-07
US201862667888P 2018-05-07 2018-05-07
US62/667,881 2018-05-07
US62/667,888 2018-05-07
US201862677474P 2018-05-29 2018-05-29
US201862667585P 2018-05-29 2018-05-29
US201862677471P 2018-05-29 2018-05-29
US62/667,585 2018-05-29
US62/677,474 2018-05-29
US62/677,471 2018-05-29
US201862703673P 2018-07-26 2018-07-26
US201862703670P 2018-07-26 2018-07-26
US62/703,670 2018-07-26
US62/703,673 2018-07-26
US201862722382P 2018-08-24 2018-08-24
US201862722388P 2018-08-24 2018-08-24
US62/722,388 2018-08-24
US62/722,382 2018-08-24
PCT/US2019/019861 WO2019169004A1 (en) 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Related Child Applications (1)

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JP2024088641A Division JP2024115558A (ja) 2018-02-27 2024-05-31 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用

Publications (4)

Publication Number Publication Date
JP2021515548A JP2021515548A (ja) 2021-06-24
JP2021515548A5 true JP2021515548A5 (https=) 2022-02-18
JPWO2019169004A5 JPWO2019169004A5 (https=) 2022-02-18
JP7498665B2 JP7498665B2 (ja) 2024-06-12

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ID=67809202

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020545159A Active JP7498665B2 (ja) 2018-02-27 2019-02-27 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用
JP2024088641A Pending JP2024115558A (ja) 2018-02-27 2024-05-31 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用

Family Applications After (1)

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JP2024088641A Pending JP2024115558A (ja) 2018-02-27 2024-05-31 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用

Country Status (14)

Country Link
US (2) US12416016B2 (https=)
EP (1) EP3768695A4 (https=)
JP (2) JP7498665B2 (https=)
KR (1) KR102848518B1 (https=)
CN (2) CN120519403A (https=)
AU (1) AU2019228504B2 (https=)
BR (1) BR112020017278A2 (https=)
CA (1) CA3091795A1 (https=)
CL (1) CL2020002201A1 (https=)
IL (1) IL276860A (https=)
MA (1) MA52087A (https=)
MX (1) MX2020008932A (https=)
SG (1) SG11202007942YA (https=)
WO (1) WO2019169004A1 (https=)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
MX2020008932A (es) * 2018-02-27 2020-10-01 Univ Pennsylvania Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
EP3884057A4 (en) * 2018-12-21 2022-09-07 The University of North Carolina at Chapel Hill OPTIMIZED GALC GENES AND EXPRESSION CASSETTE AND THEIR USE
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
PE20220014A1 (es) 2019-04-29 2022-01-11 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
CA3155016A1 (en) * 2019-10-21 2021-04-29 James M. Wilson Aav3b variants with improved production yield and liver tropism
CA3165911A1 (en) * 2020-01-03 2021-07-08 Sarepta Therapeutics, Inc. Methods for analyzing aav capsid proteins
WO2021183433A1 (en) * 2020-03-09 2021-09-16 University Of Massachusetts Gene replacement therapy for foxg1 syndrome
IL297391A (en) 2020-04-20 2022-12-01 Tenaya Therapeutics Inc An adeno-associated virus with an engineered capsid
WO2021222238A1 (en) * 2020-04-27 2021-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
IL302127A (en) 2020-10-18 2023-06-01 Univ Pennsylvania An improved adeno-associated virus (AAV) vector and its uses
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
WO2023081807A1 (en) * 2021-11-04 2023-05-11 The Trustees of the University of Pennsylvania Penn Center for Innovation Compositions and methods for reducing pcsk9 levels in a subject
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
AU2024245299A1 (en) * 2023-03-25 2025-09-25 Duke University Compositions comprising kidney-tropic aavs and methods of use thereof
WO2025250683A1 (en) * 2024-05-29 2025-12-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
WO1998046728A1 (en) 1997-04-14 1998-10-22 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
WO2004083441A2 (en) * 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Random peptide library displayed on aav vectors
EP1660970A4 (en) 2003-08-01 2007-02-14 Dna Twopointo Inc SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
CN1856576B (zh) * 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US20060246079A1 (en) 2003-11-14 2006-11-02 Morrow Phillip R Neutralizing human antibodies to anthrax toxin
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
BR112017005892A2 (pt) * 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
CA2969145A1 (en) * 2014-11-26 2016-06-02 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
HK1257801A1 (zh) 2015-10-28 2019-11-01 宾夕法尼亚州大学信托人 用於基因治疗的腺伴随病毒载体的鞘内给药
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017180854A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Novel aav8 mutant capsids and compositions containing same
IL305449B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
PE20190401A1 (es) * 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
NZ791267A (en) * 2016-08-15 2025-11-28 Genzyme Corp Methods for detecting AAV
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP7455579B2 (ja) 2017-02-28 2024-03-26 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途
MX2020008932A (es) * 2018-02-27 2020-10-01 Univ Pennsylvania Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.

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